Description

It is a question that increasingly preoccupies stakeholders from payers to biotech investors to patients: what is the value of biopharmaceuticals and how should decisions around patient access and coverage be guided by value to patients? What is needed to maintain an innovation environment and investments that produce value for patients while also considering stakeholder concerns around costs. These questions are even more pressing in the rare disease space, where the heterogeneity of disease presentation coupled with inherently small patient populations, often spread across the country and even the world, heightens the already significant challenges of drug development. This panel will convene a diverse group of stakeholders to address these questions and discuss effective mechanisms of defining and differentiating the value of rare disease therapies for the patients who rely on them.